PASADENA, Calif.—Arrowhead Research Corp., a nanomedicine
company with development programs in RNAi therapeutics and obesity, and Axolabs
GmbH, a custom research organization offering preclinical solutions and
consultancy in the field of oligonucleotide therapeutics, have entered into a
strategic alliance and master services agreement.

"This agreement continues the execution of Axolabs' strategy
to become the preeminent custom research organization in the oligonucleotide
therapeutics field," says Dr. Roland Kreutzer, managing director of Axolabs.
"We know the colleagues at Arrowhead very well. We are happy to support them in
establishing a leading role in the area of RNAi therapeutics and excited to be
chosen as their preclinical service provider."

Under the terms of the agreement, Axolabs, founded by the
principals of the former Roche Center of Excellence for RNAi Therapeutics in
Kulmbach, Germany, will provide Arrowhead and its partners with oligonucleotide
optimization, synthesis and analytics. These services include bioinformatics
for siRNA design; lead identification, optimization and characterization; as
well as siRNA synthesis and CMC-related activities.

"With multiple candidates in the clinic addressing a wide
range of indications, data demonstrating the power of RNAi as a therapeutic
modality are rapidly accumulating," says Dr. Christopher Anzalone, Arrowhead's
CEO. "Having access to Axolab's expertise in preclinical development
complements Arrowhead's capabilities well and provides us with yet another tool
for preclinical and clinical collaborations in the field. Now, with the
company's already established intellectual property position and array of
proprietary delivery technologies, including Dynamic PolyConjugates (DPC) and
RONDEL, Arrowhead is uniquely positioned to build its own pipeline of RNAi
therapeutics and provide partners entry to this promising area in a rapid and
cost-effective manner."

Arrowhead
has used a nontransgenic mouse model of chronic HBV infection of the liver to
test the efficacy of its DPC technology for delivery of anti-HBV siRNAs. These
mice produce HBsAg and HBV viral particles that are secreted into the blood. As
a prelude to successful clinical development and commercialization, Arrowhead
has secured a license granting it the exclusive right to develop, manufacture
and commercialize siRNA therapeutics against the HBV genome to treat HBV from
Alnylam Pharmaceuticals Inc.

Alnylam
President and Chief Operating Officer Barry Greene notes that there are three
known modes for overcoming the delivery hurdles that have confronted RNAi
therapeutics, liposome nanoparticles and chemistry conjugates (both Alnylam's),
and Arrowhead's Dynamic PolyConjugates approach. In addition, there is
Arrowhead's RONDEL technology—new targeted, siRNA-containing therapeutics that
employ a proprietary three-part RNAi/Oligonucleotide Nanoparticle Delivery
(RONDEL) system, the foundation of which is a cyclodextrin-containing
polymer.

"Naked"
siRNA is degraded and destroyed by nucleases in the bloodstream and is not
taken up by cells, and also causes harmful immune reactions. The RONDEL
system's cyclodextrin-containing polymers protect the
siRNA and allow it to reach its destination and perform its intended
job; for example, to stop the runaway growth of tumor cells.

Greene
points out that RNAi represents a whole new class of therapies that have the
near-term potential to transform a number of diseases. One he points to is TTR-mediated
amyloidosis (ATTR), in which Alnylam is developing ALN-TTR, a systemically
delivered RNAi therapeutic that targets the transthyretin (TTR) gene, to treat
ATTR.

ATTR
is caused by mutations in the TTR gene, which is expressed predominantly in the
liver, and results in the accumulation of pathogenic deposits of mutant and
wild-type TTR protein in multiple extra-hepatic tissues, including the
peripheral nervous system, heart, and the gastrointestinal tract.

"As
there are currently few options for patients suffering from this devastating
disease, we aim to rapidly advance our ALN-TTR program and are committed to
bringing this important medicine to ATTR patients in need," he states.